Clinical advisory board launched to boost GT-02287 development
Parkinson's therapy is designed to restore lysosomal enzyme GCase function
Gain Therapeutics has set up a clinical advisory board made up of leading Parkinson’s experts in genetics, biomarkers, and trial design to guide the advancement of GT-02287, its investigational therapy for Parkinson’s disease.
The advisory board will collaborate with Gain’s leadership to guide GT-02287 through its potential Phase 2 and Phase 3 clinical trials.
“This marks a strategic milestone for Gain Therapeutics as we advance GT-02287 into Parkinson’s patients,” Gene Mack, CEO of Gain Therapeutics, said in a company press release. “The expertise of our clinical advisory board will be instrumental in delivering a transformative treatment for Parkinson’s disease.”
The board includes Karl Kieburtz, MD, an investigator for more than 50 clinical trials in neurology; Roy Alcalay, MD, a Parkinson’s genetics and biomarkers expert; Samuel Broder, MD, former director of the National Cancer Institute; and Wilma Van de Berg, PhD, a leader in Parkinson’s biomarker research at Amsterdam UMC in the Netherlands.
GT-02287 is an orally administered, brain-penetrant small molecule that’s designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase). Genetic mutations in the GBA1 gene, which is responsible for producing GCase, are one of the most prevalent genetic risk factors associated with Parkinson’s.
GCase is crucial for breaking down and recycling fatty molecules within cells. When GBA1 is mutated, GCase’s activity is impaired, which results in harmful substances, such as misfolded proteins like alpha-synuclein and tau, accumulating, which contributes to the onset and progression of Parkinson’s disease.
What is GT-02287?
GT-02287 is designed to bind to the GCase enzyme and restore its function, which can protect nerve cells from degeneration. The therapy is meant for Parkinson’s patients with or without a GBA1 mutation.
In preclinical studies with a mouse model of Parkinson’s associated with a GBA mutation, GT-02287 administered once a day improved motor and cognitive skills, while reducing neuroinflammation and the death of dopamine-producing neurons, a critical group of nerve cells affected by Parkinson’s. The treatment also slowed or stopped the disease’s progression in Parkinson’s mouse models without GBA mutations.
A first Phase 1 clinical trial in Australia is testing GT-02287’s safety and pharmacokinetics, that is, how it moves through and is processed by the body, in 72 healthy adults up to age 64.
Results showed GT-02287 was safe, well tolerated, and detectable in the cerebrospinal fluid (CSF), the liquid that surrounds the brain and spinal cord, confirming it could reach the brain. Fluid levels of GT-02287 in trial participants were similar to those observed in rodent models treated with therapeutic doses.
Blood spot analyses revealed a 53% increase in GCase enzyme activity among those receiving GT-02287 over a placebo, demonstrating the therapy’s engagement with its target enzyme.
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